NCT02221362

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 30 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease prospectively collects data to understand clinical progression in terms of respiratory function, symptomology, genotype, biochemistry, endurance and selected subject-reported measures for 24 weeks followed by a 240 week additional observation period for up to 100 subjects.

Conditions

• Late-onset Pompe Patients Untreated or Treated With rhGAA

Timeline

• Start date: 2014-12-01
• Primary completion: 2016-09-01
• Completion: 2016-09-01
• First posted: 2014-08-20
• Last updated: 2016-10-31

Locations

24 sites across 13 countries: Australia, Brazil, Canada, Greece, Ireland, Poland, Romania, Serbia, Singapore, Slovenia, South Korea, Spain, Taiwan